SOURCE: Cellceutix

Cellceutix

August 24, 2015 07:00 ET

Cellceutix Requesting Meeting With FDA on Kevetrin for Pediatric Retinoblastoma

BEVERLY, MA--(Marketwired - August 24, 2015) - Cellceutix Corporation (OTC: CTIX) (the "Company"), a clinical stage biopharmaceutical company developing innovative therapies with oncology, dermatology, anti-inflammatory and antibiotic applications, is requesting a meeting with the U.S. Food and Drug Administration (FDA) to discuss the advancement of the Company's anti-cancer drug candidate Kevetrin for the treatment of pediatric retinoblastoma.

Retinoblastoma is the most common eye cancer affecting children usually before age 3. It is sight threatening and potentially fatal if not caught early. Occurrence is 250-300 cases per year in the U.S. The tumors arise from the retina, the light sensitive nerve tissue which lines the back of the eye. If caught early and treated it may spare the child from having the eye removed (enucleated) which occurs in late stages to prevent metastasis.

The FDA recently released draft guidance on development of drugs intended to treat rare diseases (http://www.fdanews.com/ext/resources/files/08-15/08-17-15-rarediseases.pdf?1439565970). This guidance demonstrates the FDA's flexibility in drug discovery targeting rare disease and encourages sponsors to meet with the agency to design and implement clinical studies. The FDA also encourages development of drugs and biologics to prevent and treat rare pediatric diseases through its Pediatric Disease Priority Review Voucher (PPRV) incentive program. The program is set to expire in March 2016, but could be extended under a provision in the 21stCentury Cures Act. Last week United Therapeutics Corporation agreed to sell its Priority Review Voucher to AbbVie for $350 Million. http://ir.unither.com/releasedetail.cfm?releaseid=928100

In 2013, Cellceutix conducted pre-clinical studies using human retinoblastoma cells (WERI-Rb-1) in nude mice that were implanted either subcutaneously or directly into the eye, intravitreally. Treatment with Kevetrin significantly reduced the tumor volume by more than half in the subcutaneous tumor model and showed a significant improvement in the clarity of the eye in mice treated with Kevetrin. Images from the research can be viewed at: http://cellceutix.com/kevetrin/#sthash.ODlFfr5M.HYqou6ox.dpuf.

The ongoing Phase 1 trial of Kevetrin for solid tumors has given Cellceutix a better understanding of Kevetrin's safety and mechanistic profiles making this the right time to request a meeting with the FDA. Although no retinoblastoma patient was enrolled in the Phase 1 study, the development plan for Kevetrin has always included investigating the novel compound as an important alternative to treat a rare disease to avoid such a drastic measure as enucleation (removal) of the eye of a young child. Cellceutix feels that there are many development options available in this indication, including accelerated programs such as a Pediatric Disease Priority Review Voucher, which we intend to discuss with the FDA based on the unique properties of Kevetrin.

"This is consistent with Cellceutix objectives," commented Leo Ehrlich, Chief Executive Officer at Cellceutix. "Cellceutix is now in a Phase 2 trial for Brilacidin for the prevention of oral mucositis in patients with head and neck cancer. Should the study show immunomodulatory and anti-inflammatory activity, Brilacidin will be advanced for the indications of ulcerative colitis and hidradenitis suppurativa. Likewise, a successful Phase 3 trial of Brilacidin to Treat Acute Bacterial Skin and Skin Structure Infections (ABSSSI), would lead us on our gateway path to address diabetic foot infections (DFI) with a concentrated formulation of Brilacidin that may be effective against both gram-positive and gram-negative infections required by the FDA to treat DFI. Certainly now that we have a growing understanding of Kevetrin's profile in cancer, it is appropriate for us to begin our long-standing plans to address rare pediatric diseases."

Kevetrin's mechanism and Retinoblastoma

Retinoblastoma is a malignant tumor of the developing eye that occurs in children. Retinoblastoma tumors rarely contain a mutation in the p53 gene. Tumors with wild-type p53 rely on various other mechanisms to inactivate p53. One of the most common mechanisms is increased activity of MDMX or MDM2 gene which occurs frequently in human retinoblastoma. (http://www.nature.com/nature/journal/v444/n7115/full/nature05194.html).

The antitumor activity in this type of tumor is to reactivate wild-type p53. Since many pediatric tumors maintain wild type p53 and their loss of function mutation of p53 is due to a secondary mutation affecting a growth-regulatory pathway (RB1), the reactivation of wild type p53 is therefore of particular value for the treatment of childhood tumors. The non-genotoxic activation of p53 after Kevetrin treatment is expected to result in significantly less toxicity than classical chemotherapy.

Other links:
http://www.omicsonline.org/clinical-experimental-ophthalmology-abstract.php?abstract_id=8168

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Cellceutix clinical trials on Clinicaltrials.gov:

https://clinicaltrials.gov/ct2/results?term=cellceutix&Search=Search

About Cellceutix:
Headquartered in Beverly, Massachusetts, Cellceutix is a publicly traded company under the symbol "CTIX". Cellceutix is a clinical stage biopharmaceutical company developing innovative therapies in oncology, dermatology and antimicrobial applications. Cellceutix believes it has a world-class portfolio of compounds and is now engaged in advancing its compounds and seeking strategic partnerships. Cellceutix's anti-cancer drug Kevetrin is currently in a Phase 1 clinical trial at Harvard Cancer Centers' Dana Farber Cancer Institute and Beth Israel Deaconess Medical Center. In the laboratory Kevetrin has shown to induce activation of p53, often referred to as the "Guardian Angel Gene" due to its crucial role in controlling cell mutations. Cellceutix is in a Phase 2 clinical trial with its novel compound Brilacidin-OM for the prevention of Oral Mucositis in patients with head and neck cancer. Brilacidin-OM, a defensin mimetic compound, has shown in an animal model to reduce the occurrence of severe ulcerative oral mucositis by more than 94% compared to placebo. Cellceutix's anti-psoriasis drug Prurisol is in a Phase 2 trial. Prurisol is a small molecule that acts through immune modulation and PRINS reduction. Cellceutix's lead antibiotic, Brilacidin, has completed a Phase 2b trial for Acute Bacterial Skin and Skin Structure Infections, or ABSSSI. Top-line data have shown a single dose of Brilacidin to deliver comparable clinical outcomes to the FDA-approved seven-day dosing regimen of daptomycin. Brilacidin has the potential to be a single-dose therapy for certain multi-drug resistant bacteria (Superbugs). Cellceutix has formed research collaborations with world-renowned research institutions in the United States and Europe, including MD Anderson Cancer Center, Beth Israel Deaconess Medical Center, and the University of Bologna. More information is available on the Cellceutix web site at www.cellceutix.com.

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Contact Information

  • INVESTOR AND MEDIA CONTACT:
    Cellceutix Corporation
    Leo Ehrlich
    Email contact